The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).

Start Date13 Jul 2022

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT05139810

/ CompletedPhase 3

A Phase 3 Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients With Hereditary Angioedema (HAE)

The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).

Start Date03 Dec 2021

Sponsor / Collaborator

Ionis Pharmaceuticals, Inc.

NCT04549922

/ CompletedPhase 2IIT

Antisense Therapy to Block the Kallikrein-kinin Pathway in COVID-19: A Phase II Randomized Controlled Trial

Up to 1/3 of all patients infected with COVID-19 can develop complications that require hospitalization. Severe pneumonia associated with acute respiratory distress syndrome (ARDS) is the most threatening and feared complication of COVID-19 infection, with mortality rates close to 50% in some groups.
Autopsies between these severe cases reveal severe capillary involvement, with signs of intense inflammatory changes, microvascular thrombosis, endothelial injury and abnormal tissue repair. The available evidence suggests that abnormal activation or imbalance in the counter-regulation of the kallikrein-kinin system may play a central role in a positive feedback cycle, leading to consequent diffuse microangiopathy. Blockade of the kallikrein-kinin system can therefore prevent deterioration of lung function by reducing inflammation, edema and microthrombosis.
The objective of this phase IIb study is to assess the preliminary effects on the oxygenation parameters of an antisense oligonucleotide that inhibits pre-kallikrein synthesis in patients with moderate to severe COVID-19.

Start Date19 Oct 2020

Sponsor / Collaborator

Associacao Beneficente Siria

[+2]

100 Clinical Results associated with Donidalorsen

100 Translational Medicine associated with Donidalorsen

100 Patents (Medical) associated with Donidalorsen

Literatures (Medical) associated with Donidalorsen

01 Aug 2025·ALLERGY

Patient‐Reported Outcomes in the Phase III OASIS‐HAE Study of Donidalorsen for Hereditary Angioedema

Article

Author: Treadwell, Sabrina ; Yarlas, Aaron ; Cohn, Danny M. ; Riedl, Marc A. ; Wang, Sophie ; Bordone, Laura ; Newman, Kenneth B.

ABSTRACT:

Background:

Hereditary angioedema (HAE) is a rare disease characterized by unpredictable, frequently severe swelling that negatively impacts patients' quality of life (QoL). In the phase III OASIS‐HAE study (NCT05139810), donidalorsen reduced HAE attack rate, increased disease control, and improved QoL. Here, we report further analysis of donidalorsen's impact on QoL and other patient‐reported outcomes (PROs).

Methods:

This double‐blind, placebo‐controlled study randomized patients with HAE to donidalorsen 80 mg or placebo once every 4 (Q4W) or 8 weeks (Q8W) over 24 weeks. PROs included Angioedema (AE)‐QoL Questionnaire, Angioedema Control Test (AECT), Patient Global Impression of Severity (PGIS), and Work Productivity and Activity Impairment Questionnaire plus Classroom Impairment Questions (WPAI+CIQ).

Results:

Ninety patients received donidalorsen Q4W (n = 45), donidalorsen Q8W (n = 23), or placebo (n = 22). A larger percentage of the donidalorsen Q4W group (88%) achieved clinically meaningful improvement (≥ 6‐point reduction) in AE‐QoL total score vs. placebo (45%). Both donidalorsen groups reported larger least‐squares mean (LSM) changes from baseline to week 24 vs. placebo in AE‐QoL functioning (difference: Q4W, −24.5; Q8W, −16.1), fears/shame (Q4W, −23.9; Q8W, −20.1), and nutrition (Q4W, −15.7; Q8W, −10.7) domains. Donidalorsen improved AECT scores vs. placebo (difference: Q4W, 6.0; Q8W, 4.1). A greater proportion of the donidalorsen Q4W group reported decreased disease severity vs. the placebo group (PGIS; 82% vs. 44%). Donidalorsen Q4W showed benefits vs. placebo in the presenteeism, overall work/school impairment, and activity impairment domains of the WPAI+CIQ.

Conclusions:

Donidalorsen significantly improved QoL and other PROs vs. placebo in patients with HAE.

01 Jul 2025·Journal of Allergy and Clinical Immunology-In Practice

Donidalorsen Treatment of Hereditary Angioedema in Patients Previously on Long-Term Prophylaxis

Article

Author: Lin, Tao ; Manning, Michael E ; Perego, Francesca ; Craig, Timothy ; Treadwell, Sabrina ; Newman, Kenneth B ; Jacobs, Joshua S ; Bordone, Laura ; Lumry, William R ; Yarlas, Aaron ; Riedl, Marc A ; Bernstein, Jonathan A ; Cohn, Danny M ; Banerji, Aleena ; Wedner, H James ; Gierer, Selina

BACKGROUND:

Hereditary angioedema (HAE) is a rare, potentially life-threatening disorder characterized by episodes of tissue swelling. Donidalorsen, an investigational ligand-conjugated antisense oligonucleotide, reduces plasma prekallikrein production.

OBJECTIVE:

We report an interim analysis on safety, efficacy, quality of life (QoL), and treatment preference and satisfaction from an ongoing open-label phase 3 study (OASISplus Switch cohort, NCT05392114).

METHODS:

Patients with HAE receiving stable doses (≥12 weeks) of lanadelumab, complement protein 1 inhibitor, or berotralstat switched to donidalorsen 80 mg subcutaneously every 4 weeks, using a predefined algorithm. The primary end point was the incidence and severity of treatment-emergent adverse events. Other end points included change in HAE attack rate, angioedema-QoL score, disease control (≥10 points on the Angioedema Control Test), and treatment preference and satisfaction at week 16, compared with baseline on prior treatment.

RESULTS:

A total of 65 patients were enrolled; 32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat. At cutoff, 58 were ongoing in the study (89%). Forty-five patients (70%) reported treatment-emergent adverse events; 62% were unrelated to donidalorsen. At week 16, total HAE attack rates had decreased by 62%. Hereditary angioedema attack rates decreased by 65%, 41%, and 73%, and mean angioedema-QoL scores improved by 8.4, 9.6, and 17.1 points for patients switching from lanadelumab, C1INH, and berotralstat, respectively. More patients reported well-controlled disease (93% vs 67%), and most patients preferred donidalorsen over their prior treatment, with improved treatment satisfaction.

CONCLUSIONS:

Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred donidalorsen over their prior treatment. Further analyses are planned at week 52.

01 Dec 2024·JOURNAL OF CRITICAL CARE

Antisense therapy to block the Kallikrein-kinin pathway in COVID-19: The ASKCOV randomized controlled trial

Article

Author: Buchele, Gustavo ; Gomes, Samara P C ; Santos, Maria Adelaide Dos ; Negrelli, Karina L ; Dal-Pizzol, Felipe ; Falavigna, Maicon ; de Freitas, Flávio Geraldo ; Miranda, Tamiris A ; Germano, Almir ; Gebara, Otávio ; Rosa, Regis G ; Santucci, Eliana Vieira ; Santos, Renato H N ; Zampieri, Fernando G ; Gomes, Jackeline O ; Valeis, Nanci ; Ishihara, Luciana M ; Azevedo, Luciano C P ; Cavalcanti, Alexandre B ; Berti, Isabele Ribeiro ; Machado, Flávia R ; Schettini, Daniel Almeida ; Westphal, Glauco Adrieno ; Janiszewski, Mariano ; Cohn, Danny M ; de Matos Soeiro, Alexandre ; Laranjeira, Ligia N ; de Souza Dantas, Vicente Cés ; Veiga, Viviane C ; Damiani, Lucas P

PURPOSE:

To assess the effect of antisense therapy to block kallikrein-kinin pathway in COVID-19 patients.

MATERIAL AND METHODS:

Randomized, placebo-controlled, double blind, controlled trial enrolling hospitalized COVID-19 patients that required supplementary oxygen to sustain peripheral oxygen saturation. Key exclusion criteria included use of mechanical ventilation or vasopressors, and patients with more than 10 days since symptom onset or more than 48 h of oxygen use. Patients were randomized to either one subcutaneous dose of ISIS721744, an antisense that blocks prekallikrein, or placebo. The primary outcome was the number of days alive and free of oxygen support up to 15 days (DAFOR15). Secondary endpoints included organ failure score, need and duration of mechanical ventilation up to 15 days, and all-cause mortality at 30 days. Exploratory endpoints included physiological parameters, biomarkers, and quality of life.

RESULTS:

From October 10, 2020, to December 09, 2020, 111 patients were randomized at thirteen sites in Brazil (56 to treatment and 55 to control group). Average age was 57.5 years, and most patients were male (68.5%). There were no significant differences in DAFOR15 between groups (5.9 ± 5.2 days for the intervention arm and 7.7 ± 5.1 for the control group; mean difference - 0.65, 95% confidence intervals from -2.95 to 1.36, p = 0.520).

CONCLUSION:

Antisense therapy designed to block the kallikrein-kinin pathway did not demonstrate clinical benefits in increasing days-alive without respiratory support at 15 days in patients with COVID-19 during the first wave in 2020.

CLINICALTRIALS:

GOV IDENTIFIER:

NCT04549922.

News (Medical) associated with Donidalorsen

22 Aug 2025

·pipelinereview.com

DAWNZERA™ (donidalorsen) approved in the U.S. as first and only RNA-targeted prophylactic treatment for hereditary angioedema

– DAWNZERA demonstrated significant and sustained HAE attack rate reduction and long-term disease control – – Offers longest dosing option for HAE, with dosing every 4 or 8 weeks – – Compelling profile supported by recently published switch data – – Ionis’ second independent launch in just nine months, with potential for two additional launches next year – CARLSBAD, CA, USA I August 21, 2025 I Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is the first and only RNA-targeted medicine approved for HAE, designed to target plasma prekallikrein (PKK), a key protein that activates inflammatory mediators associated with acute attacks of HAE. DAWNZERA 80mg is self-administered via subcutaneous autoinjector once every four (Q4W) or eight weeks (Q8W). HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the U.S. “DAWNZERA represents a significant advance for people living with HAE who need improved treatment options. With strong and durable efficacy, convenient administration and the longest dosing option available, we believe DAWNZERA will be the prophylactic treatment of choice for many people living with HAE. Importantly, the recently published switch data empowers patients and physicians with a roadmap for switching to DAWNZERA from other prophylactic therapies,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “At Ionis, we are dedicated to turning groundbreaking science into life-changing medicines. With the early success of our first independent launch of TRYNGOLZA® for familial chylomicronemia syndrome (FCS), and now with DAWNZERA, our second independent medicine approved in less than nine months, we are proudly delivering on that vision. To the patients, families, advocacy partners and investigators who helped make this moment a reality, we express our deepest gratitude.” The approval of DAWNZERA was based on positive results from the Phase 3 global, multicenter, randomized, double-blind, placebo-controlled OASIS-HAE study in patients with HAE. The study met its primary endpoint, with DAWNZERA Q4W significantly reducing monthly HAE attack rate by 81% compared to placebo over 24 weeks. Mean attack rate reduction increased to 87% when measured from the second dose, a key secondary endpoint. Additionally, DAWNZERA Q4W reduced moderate-to-severe HAE attacks by ~90% over 24 weeks when measured from the second dose. These results are bolstered by the ongoing OASISplus open-label extension (OLE) study, in which DAWNZERA Q8W had a similar effect as Q4W over time. DAWNZERA demonstrated 94% total mean attack rate reduction from baseline across both dosing groups after one year in the OLE. The OASISplus study also includes a switch cohort evaluating DAWNZERA Q4W in patients previously treated with lanadelumab, C1-esterase inhibitor or berotralstat for at least 12 weeks. Switching to DAWNZERA reduced mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch. A total of 84% of patients surveyed preferred DAWNZERA over their prior prophylactic treatment, citing better disease control, less time to administer and less injection site pain or reactions. Across clinical studies, DAWNZERA demonstrated a favorable safety and tolerability profile. The most common adverse reactions (incidence ≥ 5%) were injection site reactions, upper respiratory tract infection, urinary tract infection and abdominal discomfort. “As the first FDA-approved RNA-targeted therapy for HAE, DAWNZERA represents a welcome advance in therapeutic options for preventing attacks. Today’s approval gives people living with HAE and their physicians another important choice for aligning treatment with individual needs,” said Anthony J. Castaldo, CEO & chairman of the board, U.S. Hereditary Angioedema Association (HAEA) and Hereditary Angioedema International (HAEi). “People living with HAE manage this condition for all their lives, and many continue to face unpredictable, painful and dangerous breakthrough attacks even with current treatments. Durable efficacy is essential in maintaining long-term disease control,” said Marc Riedl, M.D., M.S., clinical director, U.S. HAEA Angioedema Center; University of California, San Diego; OASIS-HAE and OASISplus trial investigator. “DAWNZERA is positioned to help meet patient needs, providing substantial and sustained reduction of HAE attacks, continued improvement over time and reduced burden of treatment.” DAWNZERA will be available in the U.S. in the coming days. Ionis is committed to helping people access the medicines they are prescribed and will offer a suite of services designed to meet the unique needs of the HAE community through Ionis Every Step™. As part of Ionis Every Step, patients and healthcare providers will have access to a wide range of support and resources including dedicated support from a Patient Education Manager, assistance with the insurance approval process, information on affordability programs, access to the DAWNZERA Direct digital companion and other ongoing services and resources to help patients stay on track. Visit DAWNZERA.com for more information. Webcast Ionis will hold a webcast today at 12:15pm ET to discuss the FDA approval. Interested parties may access the webcast here . A webcast replay will be available for a limited time. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions occur, discontinue DAWNZERA and institute appropriate therapy. ADVERSE REACTIONS Most common adverse reactions (incidence ≥ 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort. Please see full Prescribing Information for DAWNZERA. About DAWNZERA™ (donidalorsen) DAWNZERA™ (donidalorsen) is approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is an RNA-targeted medicine designed to target plasma prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of HAE. For more information about DAWNZERA, visit DAWNZERA.com . About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis has marketed medicines and a leading pipeline in neurology, cardiology and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) , LinkedIn and Instagram . SOURCE: Ionis Pharmaceuticals

Clinical ResultDrug ApprovalPhase 3siRNA

22 Aug 2025

·www.biospace.com

Ionis Wins Approval For First RNA-Targeted Prophylactic for Rare Swelling Disease

iStock, Softulka The drug, for hereditary angioedema, is Ionis’ second wholly owned asset. The FDA on Thursday approved Ionis Pharmaceuticals’ donidalorsen for the prevention of disease attacks in patients with hereditary angioedema. The product will carry the brand name Dawnzera. Dawnzera is the first and so far only RNA-targeting therapy approved as a prophylactic treatment for hereditary angioedema (HAE), Ionis said in its Thursday news release . The product, which can be used in patients 12 years and above, will launch in the U.S. in the coming days. “We see Dawnzera’s pro superior to current prophylactic options, with less frequent dosing and at-home auto-injector dosing being further differentiators,” William Blair analysts told investors in a Thursday note, adding that Dawnzera marks Ionis’ second wholly owned asset. “We view Dawnzera as a highly competitive HAE prophylactic.” Data from the OASIS-HAE study backed Thursday’s approval. Results published in the New England Journal of Medicine in May 2024 showed that over 24 weeks of follow-up, the RNA-targeted therapy lowered the monthly HAE attack rate by 81% versus placebo. Dawnzera also demonstrated an approximately 90% reduction in moderate-to-severe HAE attacks over 24 weeks when measured from the second dose, and significantly improved patients’ quality of life. Ionis filed data from the Phase III OASISplus open-label extension study to bolster its application. According to Thursday’s release, Dawnzera treatment resulted in a 94% drop in the total mean attack rate from baseline at one year of follow-up. Meanwhile, a readout last month demonstrated that patients who switched to the RNA-targeting therapy from other prophylactics did so without experiencing spikes in attacks. In a survey, 84% of patients indicated that they preferred Dawnzera over their previous prophylactic. Administered subcutaneously every four weeks, Dawnzera is a conjugated antisense oligonucleotide that targets the prekallikrein mRNA, causing its destruction. This reduces overall levels of PKK, a protein that plays a role in swelling and pain attacks in HAE. Dawnzera does not come with a boxed warning, though its label has precautions for hypersensitivity reactions such as anaphylaxis. Dawnzera’s approval on Thursday continues a series of history-making approvals for the rare disease space this month. Leading the pack is Jazz Pharmaceuticals, which on Aug. 6 won the FDA’s go-ahead for dordaviprone, now marketed as Modeyso, for the treatment of diffuse midline glioma with an H3 K27M mutation in patients aged one year and up who have progressed after previous interventions. Modeyso is the first drug approved for this ultrarare brain tumor. A week later, Insmed’s brensocatib won approval for non-cystic fibrosis bronchiectasis for patients 12 and over. The drug, branded as Brinsupri, is the first therapy approved for this indication and is also the first DPP1 inhibitor to win the FDA’s blessing. Finally, Precigen on Aug. 14 bagged the industry’s first immunotherapy approval for recurrent respiratory papillomatosis with zopapogene imadenovec-drba, which will carry the brand name Papzimeos.

Clinical ResultDrug ApprovalPhase 3ImmunotherapyOligonucleotide

21 Aug 2025

·www.fiercepharma.com

Ionis steps into crowded HAE market with FDA approval for Dawnzera

The FDA has signed off on three drugs in the last two months to treat the rare genetic condition hereditary angioedema. The newest to come online is Ionis Pharmaceuticals' Dawnzera. Since 2008, the FDA has approved 11 treatments for a rare genetic condition called hereditary angioedema (HAE), three of which have occurred in the last two months.The latest drug to join this cohort of medicines arrived Thursday with the FDA approval of Ionis Pharmaceuticals’ Dawnzera (donidalorsen). Despite entering a competitive, crowded market, it shouldn’t be difficult for Dawnzera to differentiate itself. As the first RNA-targeted treatment for HAE, it is a first-in-class therapy and further distinguishes itself with its dosing schedule and ability to be injected at home.The U.S. regulator has signed off on Dawnzera for prophylactic use by those age 12 years and older to reduce the likelihood of the sudden, unpredictable episodes of severe swelling in the limbs, face, abdomen and larynx that characterize HAE. The disorder, which affects 7,000 people in the U.S., can be fatal when it strikes the airways and inhibits breathing.Dawnzera was designed to target plasma prekallikrein, a protein that activates inflammatory mediators associated with acute attacks of HAE.Paving the way for approval was a phase 3 trial in which Dawnzera reduced the HAE attack rate by 81% compared to placebo over 24 weeks. The attack rate reduction increased to 87% when measured beyond the second dose of Dawnzera.Dawnzera is the third in a run of recent approvals that give patients new options to combat the disease. The products will take on Takeda’s blockbuster standard of care, an injected treatment called Takhzyro, and BioCryst Pharmaceuticals’ fast-rising oral challenger Orladeyo.“Orladeyo has less benefit than Takhzyro, with respect to reducing attack rates, and it has tolerability issues, particularly (gastrointestinal) because it is oral,” Ionis CEO Brett Monia, Ph.D., said in an interview with Fierce. “With those two drugs alone, we’re seeing that patients are switching from one drug to another trying to get a better treatment. My point is—they’re unsatisfied.”Last month, the FDA endorsed Ekterly, the first approved product from small Massachusetts biotech KalVista Pharmaceuticals, which has become the first oral on-demand medicine to address the symptoms of HAE, while others are delivered by intravenous or subcutaneous administration.Adding to the competition in the HAE prophylactic space is CSL, which scored a June FDA nod for Andembry, a self-administered prefilled pen. The treatment is dosed monthly and inhibits factor XIIa, a plasma protein that plays a key role in triggering the swelling episodes.“It’s a quite different mechanism of action, with respect to the targets its going after—we’re targeting RNA, they’re targeting a protein with an antibody,” Monia said of Andembry. “It looks like a good drug. We think donidalorsen has an overall profile that will be more attractive to many patients.” CSL will be a formidable rival as it has vast experience in the HAE space. Its drugs Berinert and Haegarda, which were approved in 2009 and 2017, respectively, were the premier HAE treatments before Takhzyro became a blockbuster in 2022.Ionis believes Dawnzera’s advantage is in its dosing regimen, which calls for patients to begin with one injection every four weeks, with an option to switch to dosing every eight weeks. In an extension study of Ionis’ phase 3 trial, eight-week dosing performed at the same level as four-week dosing, limiting swelling episodes by 94% versus placebo.Meanwhile, Takhzyro starts patients on a two-week dosing schedule, with the option to switch to every four weeks. Andembry follows a monthly dosing regimen.Ionis will charge $57,462 per dose, which works out to a $747,000 price tag for annual injections, assuming a patient stays on the four-week schedule. While the annual pricing exceeds that charged by Takeda and CSL for their prophylactic treatments, it will be significantly less for patients who shift to the eight-week dosing plan.“We’re right in line with where the market is today,” Kyle Jenne, Ionis’ chief global product strategy officer, said during a post-approval webcast Thursday. “We expect the majority of (physicians) to start patients at every four weeks and then if they’re performing well and are well controlled, they could potentially have the option of moving them to every eight weeks. We’ll have to see what that split looks like over time.” Ionis also pointed to a switch cohort in its phase 3 study that evaluated the performance of Dawnzera in patients previously treated with Takhzyro, Orladeyo or Takeda's C1 inhibitor Cinryze. In switching to Dawnzera, patients reduced their mean HAE attack rate by 62% from prior prophylactic treatment over 16 weeks, with no mean increase in breakthrough attacks observed during the switch.Additionally, a total of 84% of patients surveyed preferred Dawnzera over their prior prophylactic treatment, citing better disease control, less time to administer and less injection site pain or reactions.The switch data are not included on Dawnzera’s label but will “remain important for Ionis’s messaging on market positioning and reimbursement,” according to William Blair analyst Myles Minter, Ph.D.“Overall, we see Dawnzera’s profile as superior to current prophylactic options, with less frequent dosing and at-home auto-injector dosing being further differentiators. We also view Dawnzera’s profile as comparable to CSL Behring’s Andembry,” Minter added in an Aug. 21 note.

Phase 3Drug ApprovalClinical Result

100 Deals associated with Donidalorsen

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Hereditary Angioedema	United States	Ionis Pharmaceuticals, Inc.	21 Aug 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05392114 (OASISplus, Pubmed \| Company_Website) Manual	Phase 3	Hereditary Angioedema	65	Donidalorsen (32 switched from lanadelumab, 22 from complement protein 1 inhibitor, and 11 from berotralstat.)	zrvcvisurp(gktaoyafrt) = tdhmytgads yzwxbqtwvp (kwfcwenhvk ) View more	Positive	17 Jul 2025
NCT05139810 (OASIS-HAE, CTgov)	Phase 3	Hereditary Angioedema	91	Donidalorsen (Pooled Placebo)	wegzuptnrh(myzkyrbwjm) = jrcyojnrya mehldrbbto (lrordysafm, uwchvnivmt - uwdfixxeva) View more	-	06 Mar 2025
				Donidalorsen (Cohort A: Donidalorsen 80 mg)	wegzuptnrh(myzkyrbwjm) = bxlsluixnb mehldrbbto (lrordysafm, yojfghonsx - puzskwbtws) View more
NCT04307381 (Corporate Publications) Manual	Phase 2	Hereditary Angioedema	17	DonidalorsenDonidalorsen 80 mg SC Q4W	spfpamaswq(oyjtfhhnql) = fubjehzevi vnlkerufsg (xtozvnpikn, 7.81)	Positive	30 Oct 2024
				Donidalorsen 80 mg SC Q8WDonidalorsen 80 mg SC Q8W	tkmzpvzrth(cpaftnulcg) = yzxbmktfjd azbjkoadod (ffqkkgczsc ) View more
NCT04307381 (Phase 2 OPEN-LABEL EXTENSION OF DONIDALORSEN, ACAAI2024)	Phase 2	Hereditary Angioedema plasma prekallikrein	17	Donidalorsen 80 mg Q4W	kshcbyfmwb(tdtwfpdvbt) = ehgfmmrszc vtoibohedy (xtgmfzsuaz, 0.02 - 0.09) View more	Positive	24 Oct 2024
				Donidalorsen 80 mg Q8W	bgipuxxlys(vagdpxhmvh) = dnnuyawxbb nspfbfxvur (vuvmquqnih )
NCT05392114 (OASISplus, ACAAI2024)	Phase 3	Hereditary Angioedema kallikrein-kinin system dysregulation	83	Donidalorsen 80 mg Q4W	krhywhxjea(goysdvtchv) = Patients previously on placebo also reported improvements in AE-QoL total scores (24 points from OASIS-HAE baseline) zesycwqrum (xxqmiydlsf ) View more	Positive	24 Oct 2024
				Donidalorsen 80 mg Q8W
NCT05392114 (OASISplus, ACAAI2024)	Phase 3	Hereditary Angioedema kallikrein-kinin system dysregulation	64	Donidalorsen 80 mg every-4-weeks	sqzyiqguor(goejrjasnd) = The percentage of patients with well-controlled symptoms (AECT ≥10) increased from 67% (baseline) to 93% (Week 17) igbdwrvevc (pznapblnjr ) View more	Positive	24 Oct 2024
NCT05139810 (OASIS-HAE, Pubmed \| N Engl J Med) Manual	Phase 3	Hereditary Angioedema	90	Donidalorsen 80 mg every 4 weeks	cgwqicqlyx(vpmsirqolj) = vqcyffsaje nfrqrmmjgh (wreesyeqkb, 0.27 - 0.73) View more	Positive	04 Jul 2024
				Donidalorsen 80 mg every 8 weeks	cgwqicqlyx(vpmsirqolj) = poipihgkbe nfrqrmmjgh (wreesyeqkb, 0.65 - 1.59) View more
NCT05139810 (OASIS-HAE, Company_Website) Manual	Phase 3	Hereditary Angioedema	90	donidalorsen 80 mg Q4W	rdjszsjeyp(gxyghvsvqc) = robibtdlyj mzgzreewxl (htgyluzwjh ) Met	Positive	31 May 2024
				donidalorsen 80 mg Q8W	rdjszsjeyp(gxyghvsvqc) = bzervsjmes mzgzreewxl (htgyluzwjh ) Met
NCT04307381 (OASISplus, Company_Website) Manual	Phase 2	Hereditary Angioedema	83	Donidalorsen every four weeks (Open-Label Extension Cohort)	gtjxqsmchp(lmvpgmhces) = None etqjsevapj (kdzwegnfso )	Positive	31 May 2024
				Donidalorsen every eight weeks (Open-Label Extension Cohort)
NCT04307381 (ISIS 721744-CS3, Pubmed \| Allergy)	Phase 2	Hereditary Angioedema	17	Donidalorsen 80 mg Q4W	sctsmadksu(nuvfmiqkmu) = Mean D-dimer concentrations decreased gvjtieudzb (rpankmcezm )	Positive	01 Mar 2024
				Donidalorsen 80 mg Q8W

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Donidalorsen

Overview

Basic Info

Structure/Sequence

Related

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation